In a strategic move beyond liver-focused therapies, Intellia Therapeutics and Regeneron Pharmaceuticals have announced the expansion of their CRISPR collaboration. Following their 2016 partnership, the two companies achieved a milestone in 2021 with first-in-human gene editing results.
This expanded collaboration aims to develop in vivo CRISPR-based therapies for neurological and muscular diseases. Regeneron will contribute its proprietary adeno-associated virus vectors, while Intellia will bring its Nme2 CRISPR/Cas9 systems adapted for viral vector delivery.
Unlike traditional partnerships, no upfront payments are involved. Instead, the companies will focus on two in vivo non-liver targets initially. Intellia will lead the editing methodology, and Regeneron will handle targeted viral vector delivery.
Each company will independently lead development and commercialization for one target, with an option for the other to join in a 50/50 co-development and co-commercialization agreement. The collaboration, which began in 2016, has undergone revisions, with the latest extension until April 2024.
Their groundbreaking achievement so far includes the in vivo CRISPR/Cas9 genome editing candidate NTLA-2001 for transthyretin amyloidosis, demonstrating a reduction of serum levels of transthyretin, a key biomarkers for the disease. As Intellia and Regeneron pioneer CRISPR-based therapies beyond the liver, the scientific community anticipates potential breakthroughs in treating neurological and muscular diseases.
Check out our AAV CDMO service to expedite your gene therapy research
PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha™ 293 cell AAV high-yield platform and the π-Omega™ plasmid high-yield platform. PackGene’s mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.
Another Two-Plasmid AAV Gene Therapy Enters Clinical Trial: Gene Vector Biotechnology’s JWK001 Pioneers in Treating nAMD
On November 30, 2023, Gene Vector Biotechnology Co., Ltd. in Chengdu, China, received approval from the National Medical Products Administration's Center for Drug Evaluation (CDE) for the clinical trial of JWK001, a Class I gene therapy drug for treating neovascular...
Accelerating Gene Therapy Production: Combining next-generation AAV vectors and artificial intelligence (AI)
Recently, the France-based WIDGeT consortium emerged from a collaboration between Sanofi, WhiteLab Genomics, the TaRGeT Laboratory at Nantes University, and Institut Imagine. Scientists and companies in this consortium hope to speed up the development of gene...
First patient received dose level 2 of RGX-202, a potential one-time AAV Therapeutic for the treatment of Duchenne that includes an optimized transgene for a novel microdystrophin On track for pivotal dose determination and initiation of pivotal program in 2024...
Hodgkin lymphoma, nodular lymphocyte predominant (high-power view) Credit: Gabriel Caponetti, MD./Wikipedia/CC BY-SA 3.0The University of Pennsylvania plans to continue offering CAR-T therapy, a cancer treatment pioneered at Penn, after the Food and Drug...
Plasmids GMP Services
Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.
AAV GMP Services
Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.